Takeda Receives U.S. FDA Approval of MIMRYLO™ (rusfertide), Marking a Potential Shift in the Treatment Paradigm for Polycythemia Vera

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ADVERSE REACTIONS

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The most common (>15%) adverse reactions were injection site reactions (56%) and anemia (16%).

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USE IN SPECIFIC POPULATIONS

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To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceuticals at 1-844-662-8532 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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About MIMRYLO™

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MIMRYLO™ is a first-in-class subcutaneous treatment that mimics the action of hepcidin, a natural hormone that regulates iron homeostasis and erythrocytosis. By targeting the underlying mechanism of iron dysregulation in polycythemia vera, MIMRYLO aims to reduce excess red blood cell production and help patients maintain hematocrit control. MIMRYLO is administered once weekly via subcutaneous injection and has been generally well-tolerated in clinical trials to date. Protagonist discovered MIMRYLO and led its development through Phase 3. Takeda now has exclusive global development and commercialization rights for MIMRYLO.

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About VERIFY

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The Phase 3 VERIFY study (NCT05210790) is an ongoing, three-part, global, randomized, placebo-controlled study evaluating MIMRYLO in 293 patients with polycythemia vera over a 156-week period, with treatment extension for participants who are continuing to derive benefit from MIMRYLO beyond the 156-week treatment period. The study is evaluating the efficacy and safety of once-weekly, subcutaneously self-administered MIMRYLO in patients with uncontrolled hematocrit who are phlebotomy-dependent despite current standard of care treatment, which could include phlebotomy, hydroxyurea, interferon and/or ruxolitinib.

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The primary endpoint of the study was the proportion of patients achieving a response during Weeks 20-32, which was defined as the absence of “phlebotomy eligibility.” To meet phlebotomy eligibility, patients in the study were required to have: confirmed hematocrit ≥45% that was ≥3% higher than their baseline hematocrit value, or hematocrit ≥48%. Key secondary endpoints evaluated at Week 32 included mean number of phlebotomies, proportion of patients maintaining hematocrit <45%, mean change in fatigue score as measured by PROMIS Fatigue Short Form 8a and total symptom burden as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Version 4.0.

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All patients have completed their participation in the randomized, placebo-controlled portion of the study evaluating the efficacy and safety of MIMRYLO plus current standard of care versus placebo plus current standard of care and are now in the open-label portions of the study.

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About Polycythemia Vera (PV)

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Polycythemia vera (PV) is a chronic blood cancer characterized by the overproduction of red blood cells (erythrocytosis), which increases blood viscosity, or thickness, and can result in life threatening thrombotic events such as stroke, deep vein thrombosis and pulmonary embolism. Hematocrit is the ratio of red blood cells to the total amount of blood in the body. Achieving and maintaining controlled hematocrit levels of less than 45% is the primary treatment goal in PV to prevent thrombotic events and alleviate burdensome symptoms, including severe fatigue, difficulty in concentrating, night sweats and pruritus.

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About Takeda

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Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit

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Takeda Important Notice

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Takeda Forward-Looking Statements

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